The purpose of this study is to measure the pharmacokinetics (PK), pharmacodynamics (PD), safety, tolerability, and immunogenicity of efgartigimod PH20 SC in pediatric participants with gMG aged 2 to \<18 years. The primary goal is to confirm an appropriate dose of efgartigimod PH20 SC for pediatric patients using PK and PD results from this study. Participants will receive injections of efgartigimod PH20 SC and will be monitored for safety until the end of the study. At the end of the follow-up period, eligible participants may roll over to an open-label extension (OLE) study. The participants will be in the study for up to 14 weeks.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
12
Subcutaneous injections
Carolinas HealthCare System Neurosciences Institute - Neurology
Charlotte, North Carolina, United States
RECRUITINGNeurology Rare Disease Center
Denton, Texas, United States
RECRUITINGUniversity of Virginia
Charlottesville, Virginia, United States
RECRUITINGUZ Gent
Ghent, Belgium
RECRUITINGChildrens Hospital of Eastern Ontario
Ottawa, Canada
RECRUITINGFakultni nemocnice Brno
Brno, Czechia
RECRUITINGFakultni nemocnice Ostrava
Ostrava, Czechia
RECRUITINGAP-HM- Hôpital de La Timone
Marseille, France
RECRUITINGUniversitätsklinikum Essen
Essen, Germany
RECRUITINGHadassah Medical Center- Ein Kerem
Jerusalem, Israel
RECRUITING...and 10 more locations
Efgartigimod serum concentrations as input for compartmental, model-driven analysis to determine age and size dependency of Clearance (CL)
Time frame: Up to 12 weeks
Efgartigimod serum concentrations as input for compartmental, model-driven analysis to determine age and size dependency of Volume Distribution (Vd)
Time frame: Up to 12 weeks
Total G immunoglobulins (IgG) levels as input for pharmacokinetics (PK)/pharmacodynamics (PD) modelling analysis
Time frame: Up to 12 weeks
Anti-acetylcholine receptors antibodies (AChR-Ab) as input for pharmacokinetics (PK)/ pharmacodynamics (PD) modelling analysis
Time frame: Up to 12 weeks
Incidence of adverse events (AEs)
Time frame: Up to 14 weeks
Severity of adverse events (AEs)
Time frame: Up to 14 weeks
Incidence of serious adverse events (SAEs)
Time frame: Up to 14 weeks
Severity of serious adverse events (SAEs)
Time frame: Up to 14 weeks
Incidence of adverse events of special interest (AESI)
Time frame: Up to 14 weeks
Severity of adverse events of special interest (AESI)
Time frame: Up to 14 weeks
Efgartigimod serum concentrations
Time frame: Up to 12 weeks
Absolute values of total Immunoglobulin G (IgG) from blood samples
Time frame: Up to 12 weeks
Change from baseline values of total Immunoglobulin G (IgG) from blood samples
Time frame: Up to 12 weeks
Percentage change from baseline values of total Immunoglobulin G (IgG) from blood samples
Time frame: Up to 12 weeks
Absolute values of anti-acetylcholine receptor antibodies (AChR-Ab) from blood samples
Time frame: Up to 12 weeks
Change from baseline values of anti-acetylcholine receptor antibodies (AChR-Ab) from blood samples
Time frame: Up to 12 weeks
Percentage change from baseline values of anti-acetylcholine receptor antibodies (AChR-Ab) from blood samples
Time frame: Up to 12 weeks
Incidence of anti-drug antibodies (ADAs) against efgartigimod in serum samples
Time frame: Up to 12 weeks
Prevalence of anti-drug antibodies (ADAs) against efgartigimod in serum samples
Time frame: Up to 12 weeks
Incidence of antibodies against rHuPH20 in serum samples
Time frame: Up to 12 weeks
Prevalence of antibodies against rHuPH20 in serum samples
Time frame: Up to 12 weeks
Absolute value of total Myasthenia Gravis Activity of Daily Living (MG-ADL) score, appropriate for pediatric use
Minimum value: 0 (no impairment); Maximum value: 24 (highest impairment)
Time frame: Up to 12 weeks
Change from baseline of total Myasthenia Gravis Activity of Daily Living (MG-ADL) score, appropriate for pediatric use
Minimum value: 0 (no impairment); Maximum value: 24 (highest impairment)
Time frame: Up to 12 weeks
Absolute value of Quantitative Myasthenia Gravis (QMG) score
Minimum value: 0 (no impairment); Maximum value: 39 (most severe impairment)
Time frame: Up to 12 weeks
Change from baseline of Quantitative Myasthenia Gravis (QMG) score
Minimum value: 0 (no impairment); Maximum value: 39 (most severe impairment)
Time frame: Up to 12 weeks
Absolute value of EuroQoL 5 Dimensions Youth (EQ-5D-Y) score
Time frame: Up to 12 weeks
Change from baseline value of Neuro-QoL Pediatric Fatigue Score
Time frame: Up to 12 weeks
Change from baseline value of EuroQoL 5 Dimensions Youth (EQ-5D-Y) score
Time frame: Up to 12 weeks
Change from baseline value of Clinical Global Impression of Improvement (CGI-I)
Time frame: Up to 12 weeks
Changes in protective antibody titers to vaccines
Time frame: Up to 12 weeks
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